COMMITTEE FOR ORPHAN MEDICINAL PRODUCTS

Transcription

1 European Medicines Agency Pre-authorisation Evaluation of Medicines for Human Use Document Date: London, 1 July 2008 Doc.Ref.: EMEA/COMP/489102/2007 COMMITTEE FOR ORPHAN MEDICINAL PRODUCTS PUBLIC SUMMARY OF POSITIVE OPINION FOR ORPHAN DESIGNATION OF trifluoromethoxy)benzyloxy)-6,7-dihydro-5h-imidazo[2,1-b] [1,3] oxazine for the treatment of tuberculosis On 29 November 2007, orphan designation (EU/3/07/513) was granted by the European Commission to Dr Ulrich Granzer, Germany, for trifluoromethoxy)benzyloxy)-6,7-dihydro-5himidazo[2,1-b] [1,3] oxazine for the treatment of tuberculosis. What is tuberculosis? Tuberculosis is an infection caused by a group of bacteria called Mycobacterium. It spreads from person-to-person by inhaling the infected airborne droplets generated by sneezing and coughing. The manifestation of the disease is variable and not all patients who are infected will develop the disease. Tuberculosis is characterised by fever, cough and breathing difficulties. The infection by the tuberculosis bacteria induces formation of granulomas, which are accumulations of large numbers of cells leading to chronic inflammatory lesions. Granulomas can develop in any tissue, but occur most often in the lungs (pulmonary tuberculosis). Tuberculosis can also affect the central nervous system (meningitis), lymphatic system, genitourinary system, bones and joints. Tuberculosis is a lifethreatening condition. What are the methods of treatment available? The treatment of tuberculosis consists of the administration of a combination of antibiotics for long periods of time. Several medicinal products were authorised for the condition in the Community, at the time of submission of the application for orphan designation. Satisfactory argumentation has been submitted by the sponsor to justify the assumption that trifluoromethoxy)benzyloxy)- 6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine might be of potential significant benefit for the treatment of tuberculosis, mainly because it might improve the long-term outcome of the patients. The assumption of benefit will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status. What is the estimated number of patients affected by the condition *? Based on the information provided by the sponsor and previous knowledge of the Committee, tuberculosis was considered to affect approximately 2 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 100,000 persons in total. How is this medicinal product expected to act? trifluoromethoxy)benzyloxy)-6,7-dihydro-5h-imidazo[2,1-b] [1,3] oxazine is considered as a potential anti-mycobacterial agent. It acts by inhibiting (blocking) the building of the * Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application. 7 Westferry Circus, Canary Wharf, London, E14 4HB, UK Tel. (44-20) Fax (44-20) [email protected] European Medicines Agency, Reproduction is authorised provided the source is acknowledged

2 mycobacterial cell wall components. The cell wall protects the microorganism from the outside and from external aggressions; its disruption can kill the microorganism. What is the stage of development of this medicinal product? The effects of trifluoromethoxy)benzyloxy)-6,7-dihydro-5h-imidazo[2,1-b] [1,3] oxazine have been evaluated in experimental models. At the time of submission of the application for orphan designation, no clinical trials in patients with tuberculosis had been initiated. trifluoromethoxy)benzyloxy)-6,7-dihydro-5h-imidazo[2,1-b] [1,3] oxazine had not been authorised anywhere worldwide for tuberculosis, at the time of submission. Orphan designation of the trifluoromethoxy)benzyloxy)-6,7-dihydro-5h-imidazo[2,1-b] [1,3] oxazine had been granted in the United States for the treatment of tuberculosis. According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 10 October 2007 a positive opinion recommending the grant of the above-mentioned designation. Opinions on orphan medicinal products designations are based on the following cumulative criteria: (i) the seriousness of the condition, (ii) the existence or not of alternative methods of diagnosis, prevention or treatment and (iii) either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments. Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation. For more information: Sponsor s contact details: Dr Ulrich Granzer Forst-Kasten-Strasse 9 b Krailling Germany Telephone: Telefax: [email protected] Page 2/5

3 Patients associations contact points: TB Alert 22 Tiverton Road London NW10 3HL United Kingdom Telephone: +44 (0) Deutsche Atemwegsliga e.v. Im Prinzenpalais Burgstr Bad Lippspringe Germany Telephone: Telefax: Page 3/5

4 Translations of the active ingredient and indication in all EU languages and Norwegian and Icelandic Language Active Ingredient Indication English Treatment of tuberculosis Bulgarian (S)-2- нитро -6-(4-(трифлуорометокси) Лечение на туберкулоза бензилокси)-6,7-дихидро-5hимидазо[2,1b][1,3]oксазин Czech Léčba tuberkulózy 5H-imidazo[2,1-b][1,3]oxazín Danish Behandling af tuberkulose 5H-imidazo[2,1-b][1,3]oxazin Dutch (trifluormethoxy)benzyloxy)- Behandeling van tuberculose 6,7-dihydro- Estonian Tuberkuloosi ravi (trifluorometoksü)bensüüloksü)-6,7-dihüdro- 5H-imidaso[2,1-b][1,3]oksasiin Finnish Tuberkuloosin hoito (trifluorometoksi)bentsyylioksi)-6,7-dihydro- 5H-imidatso[2,1-b][1,3]oksatsiini French Traitement de la tuberculose (trifluorométhoxy)benzyloxy)-6,7-dihydro- German (S)-2-Nitro-6-(4- Behandlung der Tuberkulose 5H-imidazo[2,1-b][1,3]oxazin Greek (S)-2-νιτρο-6-(4-(τριφθορομεθοξυ)- Θεραπεία της φυματίωσης βενζυλοξυ)-6,7-διυδρο-5h-ιμιδαζο[2,1- b][1,3]οξαζίνη Hungarian trifluor-metoxi)benziloxi)- Tuberculosis kezelése 6,7- dihidro-5h-imidazo[2,1-b][1,3]oxazin Italian (trifluorometossi)benzilossi)- Trattamento della tubercolosi 6,7-diidro-5H-imidazo[2,1-b][1,3]ossazina Latvian (trifluorometoksi)benziloksi)- Tuberkulozes ārstēšana 6,7-dihidro-5H-imidazo[2,1-b][1,3]oksazīns Lithuanian (trifluorometoksi)benziloksi)- Tuberkuliozės gydymas 6,7-dihidro-5H-imidazo[2,1-b][1,3]oksazinas Maltese Kura tat-tuberkulosi Polish Leczenie gruźlicy (trifluorometoksy)benzyloksy)-6,7-dihydro- 5H-imidazo[2,1-b][1,3]oksazyna Portuguese (trifluorometoxi)benziloxi)- Tratamento da tuberculose 6,7-dihidro-5H-imidazo[2,1-b][1,3]oxazina Romanian (trifluorometoxi)benziloxi)- Tratamentul tuberculozei Page 4/5

5 Slovak Slovenian Spanish Swedish Norwegian Icelandic 6,7-dihidro-5H-imidazo[2,1-b][1,3]oxazínă (trifluórmetoxy)benzyloxy)- 6,7-dihydro-5H-imidazo[2,1-b][1,3]oxazín (trifluorometoksi)benziloksi)- 6,7-dihidro-5H-imidazo[2,1-b][1,3]oksazin (trifluorometoxi)benziloxi)- 6,7-dihidro-5H-imidazo[2,1-b][1,3]oxazin (trifluorometoxi)benziloxi)- 6,7-dihidro-5H-imidazo[2,1-b][1,3]oxazină (trifluorometoksy)benzyloksy)-6,7-dihydro- 5H-imidazo[2,1-b][1,3]oksazin (S)-2-nítró-6-(4-(tríflúorómetoxý)benzýloxý)- 6,7-díhýdró-5H-ímídazó[2,1-b] [1,3]oxazín Liečba tuberkulózy Zdravljenje tuberkoloze Tratamiento de la tuberculosis Behandling av tuberkulos Behandling av tuberkulose Meðferð við berklum Page 5/5